Following Type-B Meeting With FDA, Omidubicel Planned for BLA in Hematologic Malignancies

Cell therapy omidubicel, designed for patients with hematologic malignancies who are undergoing allogeneic stem cell transplant, will be submitted to the FDA for approval in 2022.

A revised analysis of manufacturing data for omidubicel (NiCord) for patients with hematologic malignancies in need of a stem cell transplant was provided to the FDA in support of a Type-B pre-biologics license application meeting, according to a press release from Gamida Cell Ltd.1

The commercial manufacturing facility needed to demonstrate comparability with the facility that manufactured omiduicel for the phase 3 trial. The company hopes to submit this application during the first half of 2022, coming slightly later than their original target of the end of 2021. Omidubicel was examined in a phase 3 trial (NCT02730299) of patients who received a stem cell transplant with omidubicel compared with the standard umbilical cord blood for patients with leukemia, lymphoma, or myelodysplastic syndrome.

“Despite the delay in timing to bring omidubicel to patients after a potential FDA approval, we are encouraged by the FDA’s reaction to our phase 3 data as the pivotal trial of omidubicel. We have gained further clarity with the FDA on the requirements for demonstrating comparability for our commercial manufacturing facility. With the FDA’s feedback in hand, we believe that we are one step closer for omidubicel to be made available to patients in need,” Julian Adams, PhD, Chief Executive Officer of Gamida Cell, said in the press release.

In May of 2020, the phase 3 trial met its primary end point of demonstrating a highly statistically significant reduction in time to neutrophil engraftment. In a later analysis, it met all 3 of its secondary end points such as achieving platelet engraftment by day 42, the proportion of patients with grade 2 or grade 3 bacterial or invasive fungal infection in the first 100 days following transplant, and the survival time following hospitalization within the first 100 days after transplant.2 

In a previous interview, Adams discussed utilizing omidubicel to expand umbilical cord stem cells to by 50-to-100 fold. The phase 3 study was conducted at 33 sites and across 4 continents. He stated that the commercial manufacturing sites continued to treat patients even after the phase 3 trial ended because there is a continuous medical need. FDA approval of the application would result in greater ease in handling treatment for these patients, he said. 

Gamida is also investigating omidubicel in a phase 1/2 clinical trial (NCT03173937) for patients with aplastic anemia. A new drug application has been filed with the FDA for the same investigational candidate under a different brand name.

References

1. Gamida Cell provides update on pre-BLA meeting with FDA for omidubicel. News Release. Gamida Cell Ltd. November 11, 2021. Accessed November 12, 2021. https://bit.ly/30mrpoU

2. Gamida Cell announces positive topline data on secondary endpoints from phase 3 clinical study of omidubicel in patients with hematologic malignancies. News Release. Gamida Cell Ltd. October 6, 2020. Accessed November 12, 2021. https://